Goring S, Taylor A, Müller K, Li TJJ, Korol EE, Levy AR, et al. Characteristics of non-randomised studies using comparisons with external controls submitted for regulatory approval in the USA and Europe: a systematic review. BMJ Open. 2019;9:e024895.
Article PubMed PubMed Central Google Scholar
Hatswell AJ, Baio G, Berlin JA, Irs A, Freemantle N. Regulatory approval of pharmaceuticals without a randomised controlled study: analysis of EMA and FDA approvals 1999-2014. BMJ Open. 2016;6:e011666.
Article PubMed PubMed Central Google Scholar
Fashoyin-Aje LA, Mehta GU, Beaver JA, Pazdur R. The on- and off-ramps of oncology accelerated approval. N Engl J Med. 2022;387:1439–42.
Grimes DA, Schulz KF. An overview of clinical research: the lay of the land. Lancet. 2002;359:57–61.
Moher D, Hopewell S, Schulz KF, Montori V, Gøtzsche PC, Devereaux PJ, et al. CONSORT 2010 explanation and elaboration: updated guidelines for reporting parallel group randomised trials. BMJ. 2010;340:c869.
Article PubMed PubMed Central Google Scholar
Pignatti F, Wilking U, Wilking N, Delgado J, Bergh J. The value of anticancer drugs—a regulatory view. Nat Rev Clin Oncol. 2021;19:207–15.
Marini BL, Goodman AM, Perissinotti AJ. The essential role of randomised controlled trials. Lancet Haematol. 2023;10:e486–e487.
Article CAS PubMed Google Scholar
van Rosmalen J, Dejardin D, van Norden Y, Löwenberg B, Lesaffre E. Including historical data in the analysis of clinical trials: Is it worth the effort? Stat Methods Med Res. 2018;27:3167–82.
Hobbs BP, Sargent DJ, Carlin BP. Commensurate priors for incorporating historical information in clinical trials using general and generalized linear models. Bayesian Anal. 2012;7:639–74.
Article PubMed PubMed Central Google Scholar
Hobbs BP, Carlin BP, Mandrekar SJ, Sargent DJ. Hierarchical commensurate and power prior models for adaptive incorporation of historical information in clinical trials. Biometrics. 2011;67:1047–56.
Article PubMed PubMed Central Google Scholar
Cheung YK, Chappell R. Sequential designs for phase I clinical trials with late-onset toxicities. Biometrics. 2000;56:1177–82.
Article CAS PubMed Google Scholar
Babb JS, Rogatko A. Patient specific dosing in a cancer phase I clinical trial. Stat Med. 2001;20:2079–90.
Article CAS PubMed Google Scholar
Ji Y, Liu P, Li Y, Bekele BN. A modified toxicity probability interval method for dose-finding trials. Clin Trials. 2010;7:653–63.
Article PubMed PubMed Central Google Scholar
Yuan Y, Hess KR, Hilsenbeck SG, Gilbert MR. Bayesian optimal interval design: a simple and well-performing design for phase I oncology trials. Clin Cancer Res. 2016;22:4291–301.
Article PubMed PubMed Central Google Scholar
Angus DC, Berry S, Lewis RJ, Al-Beidh F, Arabi Y, van Bentum-Puijk W, et al. The REMAP-CAP (randomized embedded multifactorial adaptive platform for community-acquired pneumonia) study. rationale and design. Ann Am Thorac Soc. 2020;17:879–91.
Article PubMed PubMed Central Google Scholar
Schuetze SM, Wathen JK, Lucas DR, Choy E, Samuels BL, Staddon AP, et al. SARC009: Phase 2 study of dasatinib in patients with previously treated, high-grade, advanced sarcoma. Cancer. 2016;122:868–74.
Article CAS PubMed Google Scholar
Hirakawa A, Nishikawa T, Yonemori K, Shibata T, Nakamura K, Ando M, et al. Utility of Bayesian single-arm design in new drug application for rare cancers in Japan: a case study of phase 2 trial for sarcoma. Ther Innov Regul Sci. 2018;52:334–8.
Kim ES, Herbst RS, Wistuba II, Lee JJ, Blumenschein GR Jr, Tsao A, et al. The BATTLE trial: personalizing therapy for lung cancer. Cancer Discov. 2011;1:44–53.
Article CAS PubMed PubMed Central Google Scholar
Papadimitrakopoulou V, Lee JJ, Wistuba II, Tsao AS, Fossella FV, Kalhor N, et al. The BATTLE-2 study: a biomarker-integrated targeted therapy study in previously treated patients with advanced non-small-cell lung cancer. J Clin Oncol. 2016;34:3638–47.
Article CAS PubMed PubMed Central Google Scholar
Berry DA, Dhadda S, Kanekiyo M, Li D, Swanson CJ, Irizarry M, et al. Lecanemab for patients with early Alzheimer disease: bayesian analysis of a phase 2b dose-finding randomized clinical trial. JAMA Netw Open. 2023;6:e237230.
Article PubMed PubMed Central Google Scholar
Broglio K, Meurer WJ, Durkalski V, Pauls Q, Connor J, Berry D, et al. Comparison of Bayesian vs frequentist adaptive trial design in the stroke hyperglycemia insulin network effort trial. JAMA Netw Open. 2022;5:e2211616.
Article PubMed PubMed Central Google Scholar
Muss HB, Berry DA, Cirrincione CT, Theodoulou M, Mauer AM, Kornblith AB, et al. Adjuvant chemotherapy in older women with early-stage breast cancer. N Engl J Med. 2009;360:2055–65.
Article CAS PubMed PubMed Central Google Scholar
Reis G, Silva EASM, Silva DCM, Thabane L, Milagres AC, Ferreira TS, et al. Effect of early treatment with ivermectin among patients with covid-19. N Engl J Med. 2022;386:1721–31.
Article CAS PubMed Google Scholar
Takahashi T, Yamanaka T, Seto T, Harada H, Nokihara H, Saka H, et al. Prophylactic cranial irradiation versus observation in patients with extensive-disease small-cell lung cancer: a multicentre, randomised, open-label, phase 3 trial. Lancet Oncol. 2017;18:663–71.
Nogueira RG, Jadhav AP, Haussen DC, Bonafe A, Budzik RF, Bhuva P, et al. Thrombectomy 6 to 24 h after Stroke with a mismatch between deficit and infarct. N Engl J Med. 2018;378:11–21.
Reardon MJ, Van Mieghem NM, Popma JJ, Kleiman NS, Søndergaard L, Mumtaz M, et al. Surgical or transcatheter aortic-valve replacement in intermediate-risk patients. N Engl J Med. 2017;376:1321–31.
Shah PL, Slebos D-J, Cardoso PFG, Cetti E, Voelker K, Levine B, et al. Bronchoscopic lung-volume reduction with Exhale airway stents for emphysema (EASE trial): randomised, sham-controlled, multicentre trial. Lancet. 2011;378:997–1005.
Article CAS PubMed Google Scholar
Ferreira D, Vivot A, Diemunsch P, Meyer N. Bayesian analysis from phase III trials was underused and poorly reported: a systematic review. J Clin Epidemiol. 2020;123:107–13.
Löwenberg B, Pabst T, Maertens J, Gradowska P, Biemond BJ, Spertini O, et al. Addition of lenalidomide to intensive treatment in younger and middle-aged adults with newly diagnosed AML: the HOVON-SAKK-132 trial. Blood Adv. 2021;5:1110–21.
Article PubMed PubMed Central Google Scholar
Löwenberg B, Pabst T, Maertens J, van Norden Y, Biemond BJ, Schouten HC, et al. Therapeutic value of clofarabine in younger and middle-aged (18-65 years) adults with newly diagnosed AML. Blood. 2017;129:1636–45.
Döhner H, Estey E, Grimwade D, Amadori S, Appelbaum FR, Büchner T, et al. Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel. Blood. 2017;129:424–47.
Article PubMed PubMed Central Google Scholar
Austin PC. An introduction to propensity score methods for reducing the effects of confounding in observational studies. Multivar Behav Res. 2011;46:399–424.
Plummer M rjags: Bayesian Graphical Models using MCMC R package version 4-12 (2021). https://CRAN.R-project.org/package=rjags.
R Core Team. R: A language and environment for statistical computing (2023). R Foundation for Statistical Computing, Vienna, Austria. https://www.R-project.org/.
East 6. Statistical software for the design, simulation and monitoring clinical trials. Cambridge MA: Cytel Inc.; 2020.
Kumar A, Soares H, Djulbegovic B. Are statistically non-significant findings necessarily negative? a review of all phase III randomized controlled trials in hematology conducted by NCI sponsored cooperative groups. Blood. 2005;106:293.
Qi H, Rizopoulos D, van Rosmalen J. Sample size calculation for clinical trials analyzed with the meta-analytic-predictive approach. Res Synth Methods. 2023;14:396–413.
Center for Drug Evaluation, Research. Adaptive Design Clinical Trials for Drugs and Biologics Guidance for Industry. U.S. Food and Drug Administration. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/adaptive-design-clinical-trials-drugs-and-biologics-guidance-industry (Accessed 20 Dec 2022).
Muehlemann N, Zhou T, Mukherjee R, Hossain MI, Roychoudhury S, Russek-Cohen E. A tutorial on modern Bayesian methods in clinical trials. Ther Innov Regul Sci. 2023;57:402–16.
Article PubMed PubMed Central Google Scholar
Lewis JH, Kilgore ML, Goldman DP, Trimble EL, Kaplan R, Montello MJ, et al. Participation of patients 65 years of age or older in cancer clinical trials. J Clin Oncol. 2003;21:1383–9.
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